Myelofibrosis clinical trial aims to halt cascade of symptoms

April 23, 2015
| by Tami Dennis

To say that myelofibrosis patients need more treatment options would be an understatement. The severely low platelet counts, known as thrombocytopenia, that are one of the hallmark symptoms of the disease can lead to chronic fatigue and weakness that not only damage quality of life but, ultimately, shorten life span.

Myelofibrosis patients often suffer from severely low blood platelets, or thrombocytopenia. A myelofibrosis clinical trial at City of Hope could lead to a new treatment option.

Myelofibrosis begins in the bone marrow, spurring an accumulation of malignant bone marrow cells and causing scarring that prevents the marrow from making enough healthy blood cells. As a result, the spleen and liver have to take over the cell creation function, leading to their enlargement and damage. For the patient, the result is anemia, extreme fatigue, bleeding and an increased risk of infection. Other symptoms include itching and pain.

Controlling the severely low platelet counts could counteract this cascade of symptoms and affect progression of the disease.

“City of Hope is committed to advancing the medical community’s understanding of myelofibrosis and thrombocytopenia through research studies,” said David S. Snyder, M.D., associate chair of the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope. “Finding new ways to support patients with chronic diseases is important and helps fulfill our commitment to this community.”

This type of clinical trial, and the clinical research programs in which they’re administered, can advance understanding of disease and ultimately improve patient outcomes, Snyder said.

Such trials are especially important for patients with myelofibrosis.

“Myelofibrosis can initially be a silent disease with increasing symptom severity over time,” said Srdan Verstovsek, M.D., Ph.D., principal investigator of the multi-enter study and a director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasia, Department of Leukemia, the University of Texas MD Anderson Cancer Center. “With myelofibrosis, other organs, mainly the spleen, attempt to take over the production of blood cells. The growing spleen causes significant problems for the patient because it compresses the stomach and bowels, so patients suffer malnutrition and lose weight. There remains a significant unmet medical need for patients with low platelet counts.”

This pacritinib study is important, Verstovsek says, because it’s focused on a patient population that has not been studied in any other randomized phase III myelofibrosis study. That is, it’s the first clinical study to enroll patients specifically for the low platelet counts associated with the disease.